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MDA Awards $3.5 Million in New Research Grants

The group of new and innovative research projects is designed to make an impact across all the diseases in MDA’s program

CHICAGO, Nov. 14, 2017 – The Muscular Dystrophy Association today announced 13 new research and development grants totaling $3.5 million to accelerate the development of treatments for muscular dystrophy, ALS and related life-threatening diseases. With the addition of these new grants, MDA has now awarded $13.7 million in 2017 to fund research projects aimed at unlocking the causes and mechanisms, and developing therapies for, life-threatening diseases that can take away the ability to walk, run, talk, eat and even breathe independently.

“We have seen unprecedented progress in neuromuscular disease research in the past few years, with six new drugs — four with direct ties to MDA research dollars — having been approved by the FDA to treat diseases in our program,” said MDA Senior Vice President and Scientific Director Grace Pavlath, Ph.D. “With the addition of this latest round of grants, we’ve begun funding some incredibly exciting research projects that we hope will keep the momentum going and result in more treatment options for our community.”

For the summer 2017 grants round, MDA reviewed 244 grant applications and awarded grants to 13 leading scientists conducting innovative research targeted to make an impact across all the diseases in MDA’s program.

MDA continues to fund the world’s best researchers working on the most promising science. With these new awards, the goal is to make progress in numerous areas including: elucidating the genes/mechanisms/causes of disease, screening for drug leads and further developing experimental drugs, preparing for clinical trials, supporting young investigators, and de-risking further industry investment in drug development for neuromuscular disease.

Vital research and services dollars come from generous MDA partners and supporters who organize, and donate to, community fundraising programs, including International Association of Fire Fighters (IAFF), CITGO Petroleum Corporation, Harley-Davidson Motor Company, Acosta, Albertsons Companies, Casey’s General Stores, Circle K, The Kroger Company, National Association of Letter Carriers and Dutch Bros. Coffee.

“MDA is grateful for the dedication and commitment of our partners and donors, whose generous support enables us to continue to lead the fight to find the solutions that will transform and save lives,” Pavlath added. “Together we will continue to build momentum on the pipeline of promise in research that is yielding more new drug approvals than ever before.”

The latest round of research grants was approved by MDA’s Board of Directors following careful deliberations and analysis by MDA’s Research Advisory Committee, through which leading clinicians and scientists in volunteer roles oversee the peer-review process.

Among the new research grants awarded by MDA, highlights include:

• Identifying new drugs for ALS (amyotrophic lateral sclerosis): Scientists at the Children’s Hospital in Pittsburgh are working to identify drugs that can suppress disease mechanisms for ALS caused by a mutation in the FUS gene.
• Facilitating new gene discovery in neuromuscular diseases (NMDs): Researchers at Baylor College of Medicine in Houston are working to shed light on new biological insights and advance molecular diagnostics for neuromuscular disease patients.
• Testing a potential therapy for Duchenne muscular dystrophy (DMD): Researchers at the University of Nevada School of Medicine in Reno are assessing the effects of an existing FDA-approved drug on the function of heart and skeletal muscles in a mouse model of DMD — because the drug already is FDA-approved, successful outcomes of this study could lead to rapid translation into a new class of treatments for DMD.
• Searching for drug targets in facioscapulohumeral muscular dystrophy (FSHD): Scientists at Boston Children’s Hospital in Massachusetts are using cutting-edge techniques and a novel approach to search for genetic modifiers of FSHD that could serve as concrete targets for therapy development.
• Optimizing dosing for a drug to treat mitochondrial disease: Researchers at the University of California, Davis, are working to optimize dosing in an FDA-approved drug called dimethyl fumarate, or DMF, in animal models — successful outcomes for this work could provide data to support an investigational new drug (IND) application to the FDA for a new therapy for mitochondrial myopathy.
• Testing a potential therapy for spinal-bulbar muscular atrophy (SBMA): Scientists at the University of Michigan Medical School in Ann Arbor are completing preclinical studies in a mouse model to establish the safety and efficacy of a new type of therapy to silence activity of the gene that is mutated in SBMA.

Summer 2017 grant materials:

• Powering Progress with MDA Research Dollars, by Grace Pavlath, Ph.D., MDA Senior Vice President and Scientific Director
• MDA Grants at a Glance

For more than 60 years, MDA has funded basic, clinical and translational research as it works to unlock the mechanisms of neuromuscular disease that will enable scientists and clinician-researchers to develop therapies capable of changing lives. The power of MDA’s research program lies in its big-picture approach that leverages insights and information resulting from research in one disease area to inform and advance discoveries and breakthroughs in others.

MDA has funded more than $1 billion in neuromuscular disease research since 1950, with an unprecedented four drugs approved in the last two years illustrating the vibrancy of its research program:

The U.S. Food and Drug Administration (FDA) in August 2015 approved dichlorphenamide (brand name Keveyis) to treat hyperkalemic and hypokalemic periodic paralysis. MDA provided support for the development of Keveyis beginning in the early 1990s. MDA contributed to the early-stage development of the drug, and also provided funding support for a phase 3 human clinical trial — all of which helped make this treatment possible.

The FDA in September 2016 granted accelerated approval to eteplirsen (brand name Exondys 51) to treat the most common childhood form of muscular dystrophy. Accelerated approval of the drug — the first disease-modifying drug for DMD — marked an important step forward in the development of therapies for neuromuscular diseases and marked an historic achievement for the entire DMD community. MDA has invested more than $213 million in DMD research, including support of the early development of Exondys 51.

In December 2016, the FDA granted approval to nusinersen (brand name Spinraza), the first disease-modifying drug to treat the most common genetic cause of death in infants. Approval of the drug, which is used to treat kids and adults with all forms of spinal muscular atrophy (SMA) caused by a deficiency of SMN protein, represented a landmark achievement for the entire SMA community. MDA funded early-stage development of Spinraza and, since inception, has invested more than $46 million in SMA research.

The FDA in February 2017 approved deflazacort (brand name Emflaza) to treat DMD. Approval of the drug, which is labeled for use by people 5 years or older with DMD, regardless of genetic mutation, represents continued success for MDA and the entire DMD community. MDA has a long history of supporting research and clinical study into the effects of corticosteroids, including Emflaza, in DMD, and has invested more than $1.5 million in studies to determine drug effects, mechanism of action, side effects and best dosing regimen.

Currently, MDA is funding more than 150 different research projects around the world with a combined investment of $45.6 million — dollars raised by MDA’s supporters.

About the Muscular Dystrophy Association:
MDA is leading the fight to free individuals — and the families who love them — from the harm of muscular dystrophy, ALS and related muscle-debilitating diseases that take away physical strength, independence and life. We use our collective strength to help kids and adults live longer and grow stronger by finding research breakthroughs across diseases; caring for individuals from day one; and empowering families with services and support in hometowns across America. Learn how you can fund cures, find care and champion the cause at mda.org.

Contact:
Roxan Triolo Olivas
MDA Vice President Public Relations and Community Programs
(520) 529-5305
rolivas@mdausa.org

Nicole Young
Manager
Public Relations
(312) 260-5928
nyoung@mdausa.org